WO2023129915A3 - Compositions and methods for targeted delivery to cells - Google Patents

Compositions and methods for targeted delivery to cells Download PDF

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Publication number
WO2023129915A3
WO2023129915A3 PCT/US2022/082411 US2022082411W WO2023129915A3 WO 2023129915 A3 WO2023129915 A3 WO 2023129915A3 US 2022082411 W US2022082411 W US 2022082411W WO 2023129915 A3 WO2023129915 A3 WO 2023129915A3
Authority
WO
WIPO (PCT)
Prior art keywords
cell
compositions
lipid composition
methods
lipid
Prior art date
Application number
PCT/US2022/082411
Other languages
French (fr)
Other versions
WO2023129915A2 (en
Inventor
Daniel J. Siegwart
Qiang Cheng
Sean DILLIARD
Original Assignee
The Board Of Regents Of The University Of Texas System
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by The Board Of Regents Of The University Of Texas System filed Critical The Board Of Regents Of The University Of Texas System
Publication of WO2023129915A2 publication Critical patent/WO2023129915A2/en
Publication of WO2023129915A3 publication Critical patent/WO2023129915A3/en

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle

Abstract

Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell. In some cases, the cell can be a lung cell of a particular cell type. Also described herein are pharmaceutical compositions comprising a therapeutic or prophylactic agent assembled to a lipid composition. The lipid composition can comprise an ionizable cationic lipid, and a selective organ targeting lipid. The lipid composition can further comprise a phospholipid. Further described herein are high-potency intravenous dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.
PCT/US2022/082411 2021-12-27 2022-12-27 Compositions and methods for targeted delivery to cells WO2023129915A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163294066P 2021-12-27 2021-12-27
US63/294,066 2021-12-27

Publications (2)

Publication Number Publication Date
WO2023129915A2 WO2023129915A2 (en) 2023-07-06
WO2023129915A3 true WO2023129915A3 (en) 2023-08-10

Family

ID=87000245

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2022/082411 WO2023129915A2 (en) 2021-12-27 2022-12-27 Compositions and methods for targeted delivery to cells

Country Status (1)

Country Link
WO (1) WO2023129915A2 (en)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190388562A1 (en) * 2016-12-21 2019-12-26 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery
US20210121411A1 (en) * 2018-06-19 2021-04-29 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mrna and long nucleic acids
US20210259980A1 (en) * 2018-09-04 2021-08-26 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190388562A1 (en) * 2016-12-21 2019-12-26 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery
US20210121411A1 (en) * 2018-06-19 2021-04-29 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mrna and long nucleic acids
US20210259980A1 (en) * 2018-09-04 2021-08-26 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LI CHUNHUI, YANG TONGREN, WENG YUHUA, ZHANG MENGJIE, ZHAO DEYAO, GUO SHUAI, HU BO, SHAO WANXUAN, WANG XIAOXIA, HUSSAIN ABID, LIANG: "Ionizable lipid-assisted efficient hepatic delivery of gene editing elements for oncotherapy", BIOACTIVE MATERIALS, vol. 9, 1 March 2022 (2022-03-01), pages 590 - 601, XP093041706, ISSN: 2452-199X, DOI: 10.1016/j.bioactmat.2021.05.051 *

Also Published As

Publication number Publication date
WO2023129915A2 (en) 2023-07-06

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